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Managing Non-Transfusion-Dependent Thalassemia Presents ChallengesAn essential factor in distinguishing thalassemia phenotypes is transfusion dependence. While beta-thalassemia major requires ...
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FDA Accepts Agios' sNDA for Pyrukynd in ThalassemiaThe sNDA is supported by data from two phase III studies, ENERGIZE and ENERGIZE-T, which evaluated Pyrukynd for alpha or beta-thalassemia ... its primary endpoint of hemoglobin response, as ...
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